Bifunctional Biologic Gets Orphan Drug Status for Paroxysmal Nocturnal Hemoglobinuria
KP104 is a bifunctional, first-in-class biologic that works by selectively inhibiting the alternative and terminal pathways in the complement system.
Anemias
Beti-Cel, a Gene Therapy for Beta-Thalassemia, Gets FDA Panel Support
Beti-cel is an investigational one-time gene therapy that works by adding functional copies of a modified form of the beta-globin gene into a patient’s own hematopoietic stem cells.
Bristol Myers Squibb Withdraws Luspatercept sBLA for NTD Beta Thalassemia
The application included data from the phase 2 BEYOND study.
FDA to Review Daprodustat for Anemia of Chronic Kidney Disease
The NDA is supported by data from 5 ASCEND phase 3 clinical trials.
Enrollment Paused in Trials Evaluating BCX9930 for Complement-Mediated Diseases
BCX9930 is an investigational oral Factor D inhibitor.
Daprodustat Possibly an Oral Alternative to ESAs in Incident Dialysis
In a study of incident dialysis patients, the HIF-PHI agent daprodustat demonstrated safety and efficacy similar to that of the ESA darbepoetin alfa.
Anaphylaxis Rare With IV Iron Use, But Risk Varies By Formulation
By clarifying the risk for anaphylaxis, study findings can contribute to the choice of IV iron preparations, according to investigators.
March 2022: Drug Pipeline Updates
A review of regulatory and drug development status updates.
FDA Says No to Vadadustat Approval Due to Safety Concerns
The NDA submission included data from 36 clinical trials involving over 8000 patients.
Pyrukynd Approved for Hemolytic Anemia Due to Pyruvate Kinase Deficiency
The FDA has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.
